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Completed

NCT Number: NCT03632525

Intravenous Iron in Adults With Cystic Fibrosis

This pilot interventional cohort study will examine the effects of intravenous iron in adults with cystic fibrosis and iron deficiency.

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Key information

Conditions

Cystic Fibrosis Anemia Anemia, Hypochromic Anemia, Iron-Deficiency Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Infant, Newborn, Diseases Iron Deficiencies Iron Metabolism Disorders Iron-deficiency Lung Diseases Metabolic Diseases Nutritional and Metabolic Diseases Pancreatic Diseases Respiratory Tract Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

John Radcliffe Hospital

Oxford, United Kingdom

About this study

Iron deficiency is common in adults with cystic fibrosis, and is associated with adverse outcomes. Oral iron supplementation is poorly tolerated and may be ineffective. In some centres, intravenous iron is used to correct iron deficiency, but concerns have been raised about the safety of this treatment in the setting of chronic airways infection. The investigators are therefore planning a pilot interventional cohort study examining the effects of intravenous iron in a group of adults with cystic fibrosis. Patients will be recruited in Oxford and studied prospectively over 16 weeks, with iron given at week 4. The primary focus of this single-centre pilot/feasibility study is safety, specifically in relation to infection.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Age ≥18 years with established diagnosis of cystic fibrosis
  • Iron deficiency (transferrin saturation ≤16 % or ferritin <15 μg/l, within last 4 months)

Exclusion criteria

  • Urgent (<6 weeks) need for iron supplementation
  • Active infection (currently requiring IV antibiotics)
  • Previous intravenous iron supplementation (within last 4 months)
  • Current oral iron supplementation
  • Hypersensitivity to ferric carboxymaltose
  • Active non-tuberculous mycobacterial pulmonary disease (as defined by ATS-IDSA criteria)
  • Liver failure
  • Ferritin >300 μg/l or transferrin saturation >45%
  • Pregnancy or breast feeding
  • Previous transplantation
  • Judged by member of trial team to be unlikely to comply with safety aspects of trial

Treatment and study plan

ferric carboxymaltose

Drug

Single dose of 20 mg/kg ferric carboxymaltose (maximum 1000 mg for patients with haemoglobin <14 g/dL or 500 mg for patients with haemoglobin ≥14 g/dL).

Other names: Ferrinject

Primary outcomes

  1. Incidence of new infective events during 4 weeks before intravenous iron, compared with 4 weeks after intravenous iron

    Time frame: 8 weeks

    New infective events are defined as any of:

    • New microbiological isolate on routine sputum culture (organism not cultured in 12 months prior to study)
    • Clinical infection requiring IV antibiotics (as determined by clinical team)
    • Admission to hospital for infection-related reason (as determined by clinical team)
    • Significant deterioration in lung function (>10% fall in FEV1), not otherwise explained (as determined by clinical team)

Secondary outcomes

  1. Incidence of new infective events during 12 weeks before intravenous iron, compared with 12 weeks after intravenous iron

    Time frame: 16 weeks (plus 8 weeks of retrospective data collection from notes)

    Infective events are defined as per primary outcome. Data relating to the 8 weeks prior to the 16-week prospective study period will be obtained from the medical records.

  2. Change in number of antibiotic days

    Time frame: 16 weeks

    Assessed by review of clinical notes and patient self-reporting, to determine total number of days on which the patient was treated with antibiotics

  3. Change in abundance of sputum Pseudomonas

    Time frame: 16 weeks

    Assessed by quantitative PCR

  4. Change in sputum microbiological diversity

    Time frame: 16 weeks

    Assessed by microbiota analysis (16s rRNA gene sequencing)

  5. Change in exercise capacity (shuttle walk test)

    Time frame: 16 weeks

    Standardised and validated exercise test involving exercise at progressive intensity

  6. Change in lung function (FEV1)

    Time frame: 16 weeks

    Assessed by spirometry

  7. Change in arterial oxygen saturation

    Time frame: 16 weeks

    Assessed by non-invasive pulse oximetry

  8. Change in body mass index

    Time frame: 16 weeks

    Calculated by standard formula: BMI=weight/(height squared)

  9. Change in quality of life (CFQ-R questionnaire)

    Time frame: 16 weeks

    The Cystic Fibrosis Questionnaire-Revised (CFQ-R) is a 48-item questionnaire that provides scores in twelve quality of life domains (physical functioning, vitality, emotional state, social limitations, role limitations, embarrassment, body image, eating disturbance, treatment constraints) and three symptom domains (respiratory, digestive, weight). Scores ranging from 0 to 100 are calculated for each quality of life domain, using a published method, where a higher score indicates a more favourable health status.

  10. Change in quality of life (SF-36 questionnaire)

    Time frame: 16 weeks

    The 36-item short form questionnaire (SF-36) provides scores in eight major domains of health (physical functioning, bodily pain, role limitations due to physical health problems, role limitations due to personal or emotional problems, emotional well-being, social functioning, energy/fatigue, and general health perceptions). Each is scored on a scale from 0-100, where a higher value represents a more favourable health status. The domains may be combined to provide two summary scores, namely the 'physical component summary' and the 'mental component summary', each of which is also scored from 0-100. In calculating the respective summary scores, subscales related to physical or psychological health (as appropriate) are positively weighted, according to a published method.

  11. Change in pulmonary artery pressure, assessed by echocardiography (exploratory outcome)

    Time frame: 16 weeks

    Assessed via changes in tricuspid regurgitant jet velocity

  12. Percentage of eligible patients entering and completing the study

    Time frame: 16 weeks

    Calculated based on number of eligible patients that enter and/or complete the study.

  13. Percentage of patient in whom each outcome is successfully measured

    Time frame: 16 weeks

    Calculated based on number of participating patients in whom each outcome is measured.

Sponsors and collaborators

Lead sponsor

University of Oxford

Other

Registry information

Official study title

A Pilot Trial of Intravenous Iron for the Treatment of Iron Deficiency in Adult Patients With Cystic Fibrosis

Important dates

Study start
2019
Primary completion
2020
Study completion
2021
First posted
Aug 15, 2018
Registry last updated
Sep 7, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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